The mucopolysaccharidosis treatment is expected to expand at a CAGR around 6% over 2019-2029.

Europe and North America are the most dominant regions in terms of revenue for mucopolysaccharidosis treatment.

North America is a leading region in the mucopolysaccharidosis treatment market owing to the high expenditure from the U.S. for each treatment routine and the direct presence of the leading manufacturers of mucopolysaccharidosis drugs

Increase in research activities for stem cell therapies is expected to boost the mucopolysaccharidosis treatment market.

Stem cell therapy as a treatment choice for mucopolysaccharidosis is still in the nascent stage in terms of global adoption.

With present clinical data, it seems more suitable to reconsider Hematopoietic Stem Cell Transplantation (HSCT) as a treatment option.

Lack of epidemiological data for rare diseases such as MPS -

Lack of epidemiological data on the incidence and prevalence of rare diseases obstructs the actual understanding of the extent of the burden created by rare diseases.

In such a situation, the economic load for rarest diseases is unidentified and cannot be adequately estimated, thus resulting in the poor availability of the required treatment options.

Top 3 Factors shaping the mucopolysaccharidosis treatment Market

Drug Exclusivity Provides Continuous Value Addition to Companies

Regulatory boards are likely to grant the orphan drug designation to drugs proposed for the treatment of rare diseases or conditions. The orphan drug designation provides market exclusivity for a concerned drug type, which needs to be generated before submitting an application for marketing approval. Mucopolysaccharidosis treatment falls under the rare disease category due to which emerging market players are adopting various strategies to boost the sales of their products.

The orphan drug designation for mucopolysaccharidosis treatment provides prospects such as financial backing for clinical trial costs, tax benefits, and FDA user fee benefits. Majority of the companies present in the mucopolysaccharidosis treatment market are directly involved in receiving drug exclusivity, which is expected to boost the revenue generation as well as result in a significant increase in competition.

Cost of Treatment is Hampering its Adoption

The cost of the treatment of rare diseases, such as mucopolysaccharidosis, is high irrespective of whether a patient opts for a standard treatment such as ERT or emerging and promising technologies such as stem cell therapies. The high cost of mucopolysaccharidosis treatment significantly indirectly affects the businesses of manufacturers due to factors such as potential patient count, sluggish awareness, and lack of epidemiological data for better calculations.

The real cost of Mucopolysaccharidosis treatment differs by region due to factors such as socioeconomic conditions, birth rate, R&D investments, and healthcare expenditure, among others, which also influence the treatment cost in each region. The cost of mucopolysaccharidosis treatment in the North America region has been found to be significantly higher than the global average treatment cost.

Emerging Technologies are Contributing to Introduction of New MPS Treatment Options

A number of emerging treatments and therapies are currently in the development phase for MPS treatment. Nowadays, companies are involved in discovering the next major drug instead of waiting to benefit from the patent expiration of an exclusive drug type, which is proving to be an effective strategy. The existing drug types are facing competition from emerging candidates.

The development of mucopolysaccharidosis treatment by companies such as Abeona Therapeutics, ArmaGen, and others is likely to provide a more progressive structure for the mucopolysaccharidosis treatment market in the coming years. For instance, on January 23, 2018, a Swedish Orphan Biovitrum AB received FDA acceptance based on its investigational new drug application for SOBI003, which has been issued for mucopolysaccharidosis treatment.

Mucopolysaccharidosis treatment Market Structure Analysis

The current global Mucopolysaccharidosis treatment market is controlled by four major companies: Takeda Pharmaceutical Company Limited, Sanofi S.A., BioMarin, and Ultragenyx Pharmaceutical Inc.

A number of companies have products in the clinical phases with an orphan drug exclusivity

The Mucopolysaccharidosis treatment market is highly consolidated with very few market players, however, the emergence of new manufacturers is slow due the requirement of a long time period for the approval of drugs as well as lack of resources

A recent market study published by PMR– “Mucopolysaccharidosis Treatment Market: Global Industry Analysis 2014-2018 & Forecast, 2019-2029” consists of a comprehensive assessment of the most important market dynamics. On conducting a thorough research of the historic as well as current growth parameters of the mucopolysaccharidosis treatment market, the growth prospects are obtained with maximum precision.

The mucopolysaccharidosis treatment market report features the unique and salient factors that are likely to significantly impact the development of the mucopolysaccharidosis treatment market during the forecast period. It can help market players to modify their manufacturing and marketing strategies to envisage maximum growth in the mucopolysaccharidosis treatment market in the upcoming years. The report provides detailed information about the current and future growth prospects of the mucopolysaccharidosis treatment market in the most comprehensive manner for the better understanding of readers.

Chapter 1 – Executive Summary

The mucopolysaccharidosis treatment market report commences with an executive summary of the key findings and key statistics of the Mucopolysaccharidosis Treatment market. It also includes the market value (US$ million) estimates of the leading segments of the mucopolysaccharidosis treatment market.

Chapter 2 – Market Overview

Readers can find detailed market taxonomy which highlights the inclusions and exclusions for the subject. The definition of mucopolysaccharidosis treatment market is included in this chapter, which helps in understanding the basic information about the concerned mucopolysaccharidosis treatment market, which helps the reader understand the scope of the mucopolysaccharidosis treatment market report.

Chapter 3 – Key Trends

This section explains about the key trends followed by the manufacturer and consumer in mucopolysaccharidosis treatment market. This section helps reader to understand the both supply-side and demand-side trend impacting the growth of mucopolysaccharidosis treatment market.

Chapter 4 – Key Success Factors

This chapter highlights the key success factors of the mucopolysaccharidosis treatment market, which include regulatory scenario, pipeline analysis, snapshot of developments for mucopolysaccharidosis Type 3, rare disease framework and designed designation for each treatment present.

This section explain the global market analysis and forecast for the mucopolysaccharidosis treatment market. It also highlights the incremental opportunity for the mucopolysaccharidosis treatment market along with the absolute dollar opportunity for every year between the forecast period of 2019-2029.

Chapter 6 – Market Background

This chapter explains the key macro-economic factors that are expected to influence the growth of the mucopolysaccharidosis treatment market over the forecast period. Along with macroeconomic factors, this section also highlights the opportunity analysis for the mucopolysaccharidosis treatment market. This chapter also highlights the key dynamics of the mucopolysaccharidosis treatment market, which include the drivers and restraints.

Based on the Treatment Type, the mucopolysaccharidosis treatment market is segmented into Enzyme Replacement Therapies and Stem Cell Therapies. Stem Cell Therapies is further segmented into Bone Marrow Transplantation and Umbilical Cord Blood Transplantation. In this chapter, readers can find a detailed analysis of the mucopolysaccharidosis treatment market by different Treatment Type and their expected growth over the forecast period.

Based on the Type of MPS, the mucopolysaccharidosis treatment market is segmented into MPS I, MPS II, MPS IV A, MPS VI and MPS VII. This section helps readers understand the prevalence of different Type of MPS in the mucopolysaccharidosis treatment market over the forecast period.

Based on end user, the mucopolysaccharidosis treatment market is segmented into Hospital, Specialty Clinics, Medical Research Centers and Home-infusion. In this chapter, readers can also understand the market attractive analysis based on the end user.

This chapter explains how the mucopolysaccharidosis treatment market will grow across various geographic regions such as North America, Latin America, Europe, East Asia, South Asia, Oceania and the Middle East & Africa (MEA).

This chapter includes a detailed analysis of the growth of the North America mucopolysaccharidosis treatment market along with a country-wise assessment, which includes the U.S. and Canada. Readers can also find the key takeaways of this region, and market growth based on treatment type, type of MPS, end user and country of mucopolysaccharidosis treatment in the North America region.

This chapter contains a snapshot of the Latin America mucopolysaccharidosis treatment market. It includes the growth prospects of the mucopolysaccharidosis treatment market in the leading LATAM countries such as Brazil, Mexico, Argentina and the rest of the Latin America region.

The important growth prospects of the mucopolysaccharidosis treatment market based on treatment type, type of MPS and end user in several European countries, such as the U.K., Germany, France, Italy, Spain, Russia and the rest of Western Europe, is included in this chapter.

This chapter highlights the growth of the mucopolysaccharidosis treatment market in Eastern Europe by focusing on China, Japan and South Korea. This section also helps readers understand the key factors that are responsible for the growth of the mucopolysaccharidosis treatment market in East Asia.

India, Indonesia, Thailand , Malaysia are the leading countries in the South Asia region that are the prime subjects of assessment to obtain the growth prospects of the South Asia mucopolysaccharidosis treatment market in this chapter. Readers can find detailed information about the growth parameters of the South Asia mucopolysaccharidosis treatment market during the period 2019-2029.

Readers can find important factors that can significantly impact the growth of the mucopolysaccharidosis treatment market in Australia and New Zealand during the forecast period based on the market segmentation.

This chapter provides information about the growth of the mucopolysaccharidosis treatment market in the major countries of the MEA region, such as GCC Countries and South Africa, during the period 2019-2029.

Chapter 18 – Market Structure Analysis

This section explains the tier structure for global mucopolysaccharidosis treatment market which helps reader to understand the percent share of market cover by tier 1, tier 2, and tier 3 players in the mucopolysaccharidosis treatment market. This section also explains the company share analysis for mucopolysaccharidosis treatment market which helps readers to understand the market share taken by key players available in the market.

Chapter 19 – Competition Landscape

In this chapter, readers can find a comprehensive list of all the leading manufacturers in the mucopolysaccharidosis treatment market, along with detailed information about each company, which includes the company overview, revenue shares, strategic overview, and recent company developments. Some of the players featured in the mucopolysaccharidosis treatment market report are Sanofi S.A., Shire, BioMarin Pharmaceutical Inc., Ultragenyx Pharmaceutical Inc., Sarepta Therapeutics, Abeona Therapeutics, Inc., ArmaGen, Eloxx Pharmaceuticals, Esteve, Immusoft Corporation, Inventiva.

Chapter 20 – Assumptions and Acronyms

This chapter includes a list of acronyms and assumptions that provide a base to the information and statistics included in the report.

Chapter 21 – Research Methodology

This chapter helps readers understand the research methodology followed to obtain various conclusions and important qualitative information & quantitative information about the mucopolysaccharidosis treatment market.